A study to evaluate the long-term clinical safety and efficacy of subcutaneously administered C1-esterase inhibitor in the prevention of hereditary angioedema

Phase 1

• Conditions: Hereditary Angioedema Types I and II; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; MedDRA version: 17.1Level: PTClassification code 10019860Term: Hereditary angioedemaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders

• Registration Number: EUCTR2014-001054-42-ES
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-01-05
• Last Update Posted: 9 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

-Males or females aged 6 years or older.
-A confirmed diagnosis of HAE type I or II.
-HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.
-For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit.
Are the trial subjects under 18? yes
Number of subjects for this age range: 11
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 88
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 11

Exclusion Criteria

-Incurable malignancies.
-Any clinical condition that will interfere with the evaluation of C1-INH therapy.
-Clinically significant history of poor response to C1-esterase therapy for the management of HAE.
-Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.
-Inability to have HAE managed pharmacologically with on-demand treatment.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.;Secondary Objective: -To further characterize the clinical safety of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.<br><br>-To characterize the clinical efficacy of subcutaneously administered C1-INH in the long-term prophylactic treatment of HAE.;Primary end point(s): The person-time incidence rates of specified safety events.;Timepoint(s) of evaluation of this end point: During the treatment phase, up to 52 weeks.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Percentage of subjects with SAEs or other specified safety events.<br>- Percentage of C1-INH injections resulting in solicited AEs (injection site reactions).<br>- Percentage of subjects with at least 1 solicited AE (injection site reaction).<br>- Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus.<br>- Percentage of subjects who experience < 1 HAE attack per 4-week period.<br>- Percentage of subjects with a ? 50% reduction in the time-normalized number of HAE attacks.;Timepoint(s) of evaluation of this end point: - During the treatment phase, up to 52 weeks.<br>- During the treatment phase, up to 52 weeks.<br>- During the treatment phase, up to 52 weeks.<br>- From baseline through the treatment phase, up to 52 weeks.<br>- During the treatment phase, up to 52 weeks.<br>- From baseline through the treatment phase, up to 52 weeks.